FDA GRANTS CHELSEA'S DROXIDOPA ORPHAN DRUG DESIGNATION

Chelsea Therapeutics International announced that the FDA has granted orphan drug designation to Droxidopa for the treatment of symptomatic neurogenic orthostatic hypotension (NOH) in patients with primary autonomic failure, a group of diseases that includes Parkinson's disease, pure autonomic failure and multiple systems atrophy.

Symptomatic NOH is a neurogenic disorder resulting from a deficient release of norepinephrine, the neurotransmitter used by sympathetic autonomic nerves to send signals to the blood vessels and the heart.

The Orphan Drug Act provides for economic incentives to encourage the development of drugs for diseases affecting fewer than 200,000 people in the U.S., according to the company. Orphan drug designation will entitle Chelsea to seven years of market exclusivity for Droxidopa in the treatment of symptomatic NOH. Additional benefits include tax credits related to clinical trial expenses, a possible exemption from the FDA-user fee and assistance in clinical trial protocol design.

Chelsea plans to initiate a double-blind pivotal Phase III trial comparing Droxidopa to placebo at multiple sites in the U.S. and Europe during the second half of this year. The trial is intended to assess the safety and efficacy of Droxidopa in patients suffering from symptomatic NOH with the primary efficacy endpoint being defined as improvement in orthostatic blood pressure over time.

The company also filed an application for orphan designation for the treatment of symptomatic NOH in patients with primary autonomic failure with the European Medicines Agency.